JPWO2020097548A5 - - Google Patents
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- Publication number
- JPWO2020097548A5 JPWO2020097548A5 JP2021525113A JP2021525113A JPWO2020097548A5 JP WO2020097548 A5 JPWO2020097548 A5 JP WO2020097548A5 JP 2021525113 A JP2021525113 A JP 2021525113A JP 2021525113 A JP2021525113 A JP 2021525113A JP WO2020097548 A5 JPWO2020097548 A5 JP WO2020097548A5
- Authority
- JP
- Japan
- Prior art keywords
- lipid
- compound
- nucleic acid
- lipid nanoparticles
- dma
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 150000002632 lipids Chemical class 0.000 claims 34
- 239000002105 nanoparticle Substances 0.000 claims 24
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 14
- 150000001875 compounds Chemical class 0.000 claims 14
- 108020004707 nucleic acids Proteins 0.000 claims 12
- 102000039446 nucleic acids Human genes 0.000 claims 12
- 150000007523 nucleic acids Chemical class 0.000 claims 12
- 235000012000 cholesterol Nutrition 0.000 claims 7
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 6
- 238000000034 method Methods 0.000 claims 6
- 239000000203 mixture Substances 0.000 claims 6
- 201000010099 disease Diseases 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 150000003839 salts Chemical class 0.000 claims 4
- 229940125898 compound 5 Drugs 0.000 claims 3
- 229920001184 polypeptide Polymers 0.000 claims 3
- 102000004196 processed proteins & peptides Human genes 0.000 claims 3
- CPELXLSAUQHCOX-UHFFFAOYSA-M Bromide Chemical compound [Br-] CPELXLSAUQHCOX-UHFFFAOYSA-M 0.000 claims 2
- KZBUYRJDOAKODT-UHFFFAOYSA-N Chlorine Chemical compound ClCl KZBUYRJDOAKODT-UHFFFAOYSA-N 0.000 claims 2
- 108020004459 Small interfering RNA Proteins 0.000 claims 2
- 125000002091 cationic group Chemical group 0.000 claims 2
- 229940125782 compound 2 Drugs 0.000 claims 2
- 239000003937 drug carrier Substances 0.000 claims 2
- 230000009395 genetic defect Effects 0.000 claims 2
- 108020004999 messenger RNA Proteins 0.000 claims 2
- 230000002018 overexpression Effects 0.000 claims 2
- 238000011321 prophylaxis Methods 0.000 claims 2
- 230000001225 therapeutic effect Effects 0.000 claims 2
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 claims 1
- 208000008425 Protein deficiency Diseases 0.000 claims 1
- DHKHKXVYLBGOIT-UHFFFAOYSA-N acetaldehyde Diethyl Acetal Natural products CCOC(C)OCC DHKHKXVYLBGOIT-UHFFFAOYSA-N 0.000 claims 1
- 125000002777 acetyl group Chemical class [H]C([H])([H])C(*)=O 0.000 claims 1
- 230000004071 biological effect Effects 0.000 claims 1
- 229940125773 compound 10 Drugs 0.000 claims 1
- 230000007812 deficiency Effects 0.000 claims 1
- 230000014509 gene expression Effects 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- ZLVXBBHTMQJRSX-VMGNSXQWSA-N jdtic Chemical compound C1([C@]2(C)CCN(C[C@@H]2C)C[C@H](C(C)C)NC(=O)[C@@H]2NCC3=CC(O)=CC=C3C2)=CC=CC(O)=C1 ZLVXBBHTMQJRSX-VMGNSXQWSA-N 0.000 claims 1
- 230000001590 oxidative effect Effects 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862758055P | 2018-11-09 | 2018-11-09 | |
| US62/758,055 | 2018-11-09 | ||
| PCT/US2019/060595 WO2020097548A1 (en) | 2018-11-09 | 2019-11-08 | Lipid nanoparticle formulations |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2022506955A JP2022506955A (ja) | 2022-01-17 |
| JP2022506955A5 JP2022506955A5 (https=) | 2022-11-07 |
| JPWO2020097548A5 true JPWO2020097548A5 (https=) | 2022-11-07 |
| JP7506670B2 JP7506670B2 (ja) | 2024-06-26 |
Family
ID=70612210
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021525113A Active JP7506670B2 (ja) | 2018-11-09 | 2019-11-08 | 脂質ナノ粒子製剤 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220001029A1 (https=) |
| EP (1) | EP3877522A4 (https=) |
| JP (1) | JP7506670B2 (https=) |
| CN (1) | CN113728101A (https=) |
| AU (1) | AU2019377131A1 (https=) |
| CA (1) | CA3118559A1 (https=) |
| WO (1) | WO2020097548A1 (https=) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN113166783B (zh) | 2018-10-09 | 2024-10-11 | 不列颠哥伦比亚大学 | 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法 |
| KR20210105889A (ko) | 2018-11-09 | 2021-08-27 | 아뷰터스 바이오파마 코포레이션 | 지질 나노입자 제제 |
| CN114729376A (zh) | 2019-09-23 | 2022-07-08 | 欧米茄治疗公司 | 用于调节肝细胞核因子4α(HNF4α)基因表达的组合物和方法 |
| AU2020355000A1 (en) | 2019-09-23 | 2022-03-17 | Omega Therapeutics, Inc. | Compositions and methods for modulating apolipoprotein B (APOB) gene expression |
| CN116096886A (zh) | 2020-03-11 | 2023-05-09 | 欧米茄治疗公司 | 用于调节叉头框p3(foxp3)基因表达的组合物和方法 |
| EP4132478A1 (en) | 2020-04-09 | 2023-02-15 | Finncure Oy | Mimetic nanoparticles for preventing the spreading and lowering the infection rate of novel coronaviruses |
| US12194157B2 (en) | 2020-04-09 | 2025-01-14 | Finncure Oy | Carrier for targeted delivery to a host |
| CZ310613B6 (cs) | 2020-09-23 | 2026-01-28 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Lipidoidy pro transfekci nukleových kyselin a jejich použití |
| JP2023546175A (ja) | 2020-10-14 | 2023-11-01 | ジョージ・メイソン・リサーチ・ファウンデーション・インコーポレイテッド | 脂質ナノ粒子製造の方法及びそれに由来する組成物 |
| CN112980878B (zh) * | 2021-02-04 | 2023-03-31 | 中国农业科学院兰州兽医研究所 | Hdac8基因敲除的bhk-21细胞系及其构建方法和应用 |
| CN112877275B (zh) * | 2021-02-04 | 2023-03-31 | 中国农业科学院兰州兽医研究所 | Hdac2基因敲除的bhk-21细胞系及其构建方法和应用 |
| EP4367242A2 (en) | 2021-07-07 | 2024-05-15 | Omega Therapeutics, Inc. | Compositions and methods for modulating secreted frizzled receptor protein 1 (sfrp1) gene expression |
| CZ310443B6 (cs) | 2021-07-19 | 2025-06-25 | Ústav organické chemie a biochemie AV ČR, v. v. i. | Cyklohexanové lipidoidy pro transfekci nukleových kyselin a jejich použití |
| KR20230053998A (ko) | 2021-10-15 | 2023-04-24 | (주)지노믹트리 | mRNA 발현을 위한 유전자 구조체 |
| CN118804743A (zh) * | 2022-03-04 | 2024-10-18 | 益杰立科(上海)生物科技有限公司 | 靶向递送系统和方法 |
| WO2024009316A1 (en) * | 2022-07-04 | 2024-01-11 | Bharat Biotech International Limited | Cationic lipid based composition, formulation and use for nucleic acid vaccine delivery and preparation thereof |
| WO2024243438A2 (en) | 2023-05-23 | 2024-11-28 | Omega Therapeutics, Inc. | Compositions and methods for reducing cxcl9, cxcl10, and cxcl11 gene expression |
| EP4722368A1 (en) | 2023-05-26 | 2026-04-08 | Samsung Biologics Co., Ltd. | Novel nucleic acid construct comprising poly(a) tail having secondary or tertiary structure and uses thereof |
| KR20250018367A (ko) | 2023-07-28 | 2025-02-05 | 한국과학기술원 | 히스톤 코드 리더 도메인을 포함하는 폴리펩티드 및 이의 용도 |
| WO2025144938A1 (en) | 2023-12-26 | 2025-07-03 | Emmune, Inc. | Systems for nucleic acid transfer |
| WO2025166323A2 (en) | 2024-02-02 | 2025-08-07 | Editas Medicine, Inc. | Crispr-related methods and compositions targeting lipoprotein (a) expression |
| WO2025210520A1 (en) * | 2024-04-04 | 2025-10-09 | Genevant Sciences Gmbh | Lipid nanoparticles for inducing an immunological response |
| WO2025213138A1 (en) | 2024-04-05 | 2025-10-09 | Editas Medicine, Inc. | Crispr/rna-guided nuclease related methods and compositions for treating primary open angle glaucoma |
| CN120037202B (zh) * | 2025-01-14 | 2025-12-26 | 浙江大学 | 一种基于连接处含有分支结构的阳离子脂质的脂质纳米颗粒及其应用 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2621480B1 (en) * | 2010-09-30 | 2018-08-15 | Sirna Therapeutics, Inc. | Low molecular weight cationic lipids for oligonucleotide delivery |
| JP2013545727A (ja) * | 2010-10-21 | 2013-12-26 | メルク・シャープ・アンド・ドーム・コーポレーション | オリゴヌクレオチド送達用の新規低分子量カチオン性脂質 |
| US9126966B2 (en) * | 2011-08-31 | 2015-09-08 | Protiva Biotherapeutics, Inc. | Cationic lipids and methods of use thereof |
| SG11201405157PA (en) * | 2012-02-24 | 2014-10-30 | Protiva Biotherapeutics Inc | Trialkyl cationic lipids and methods of use thereof |
| AU2013249548A1 (en) * | 2012-04-19 | 2014-11-06 | Sirna Therapeutics, Inc. | Novel diester and triester based low molecular weight, biodegradable cationic lipids for oligonucleotide delivery |
| WO2015011633A1 (en) * | 2013-07-23 | 2015-01-29 | Protiva Biotherapeutics, Inc. | Compositions and methods for delivering messenger rna |
| BR112017006679A2 (pt) | 2014-10-02 | 2017-12-26 | Protiva Biotherapeutics Inc | moléculas, composição, partícula, composição farmacêutica, métodos para silenciar a expressão de um gene, usos de uma partícula, métodos para melhorar um ou mais sintomas, métodos para tratar uma infecção, usos de uma composição, método para inibir a replicação do vírus da hepatite d |
| AU2017379901B2 (en) | 2016-12-21 | 2024-02-15 | Arbutus Biopharma Corporation | Methods for ameliorating infusion reactions |
| KR20210105889A (ko) * | 2018-11-09 | 2021-08-27 | 아뷰터스 바이오파마 코포레이션 | 지질 나노입자 제제 |
-
2019
- 2019-11-08 US US17/291,960 patent/US20220001029A1/en not_active Abandoned
- 2019-11-08 JP JP2021525113A patent/JP7506670B2/ja active Active
- 2019-11-08 EP EP19882056.5A patent/EP3877522A4/en not_active Withdrawn
- 2019-11-08 AU AU2019377131A patent/AU2019377131A1/en active Pending
- 2019-11-08 WO PCT/US2019/060595 patent/WO2020097548A1/en not_active Ceased
- 2019-11-08 CA CA3118559A patent/CA3118559A1/en active Pending
- 2019-11-08 CN CN201980088382.9A patent/CN113728101A/zh active Pending
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